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EXTON, PA, March 18, 2025 (GLOBE NEWSWIRE) -- Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare and debilitating blood disorder that affects approximately 8,000 to 10,000 people in North America...

EXTON, PA, June 26, 2024 (GLOBE NEWSWIRE) -- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disorder in which red blood cells prematurely break apart, a process known as hemolysis....

EXTON, PA, June 10, 2024 (GLOBE NEWSWIRE) -- Spherix Global Insights, a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry,...

The United States Food and Drug Administration (FDA) has awarded orphan drug designation (ODD) to NM5072 for the treatment of anemia in PNH patients.The ODD provides NM5072 with a range of benefits,...

FDA grants orphan drug designation to Ruxoprubart (NM8074) for the treatment of PNH.Ruxoprubart is an Anti-Bb antibody that selectively blocks the alternative pathway while maintaining the intactness...

The USAN Council has officially adopted "Ruxoprubart (Ruk”soe proo' bart)" as the generic name for NM8074, marking a significant milestone in the drug development process.Ruxoprubart is an...

The Phase I healthy trial revealed that NM8074 effectively blocked the alternative pathway (AP) and AP-mediated C3b deposition without affecting the classical pathway (CP). Importantly, NM8074 did not...

Phase I Clinical Trial in healthy subjects shows that NM3086 was safe and well tolerated at all doses (0.1 to 20 mg/kg) in six cohorts. Total AP inhibition was achieved through all cohorts and the...

—Berotralstat approval/launch timelines in U.S., Japan, EU remain on track— —Early clinical data supports BCX9930 as oral monotherapy for complement-mediated diseases— —Patient dosing underway in...

—Phase 1 data showed rapid, sustained and >95% inhibition of alternative pathway of complement system with 100 mg of oral BCX9930 BID— —BCX9930 safe and generally well tolerated— —Part 1 SAD and...